Félix Ratjen
A5017024281- Cystic Fibrosis Research Advances
- Neonatal Respiratory Health Research
- Tracheal and airway disorders
- Asthma and respiratory diseases
- Chronic Obstructive Pulmonary Disease (COPD) Research
- Inhalation and Respiratory Drug Delivery
- Pediatric health and respiratory diseases
- Respiratory Support and Mechanisms
- Vascular Anomalies and Treatments
- Atomic and Subatomic Physics Research
- Respiratory viral infections research
- Pneumonia and Respiratory Infections
- Congenital Diaphragmatic Hernia Studies
- Antibiotic Resistance in Bacteria
- Neuroscience of respiration and sleep
- Delphi Technique in Research
- Bacterial biofilms and quorum sensing
- Interstitial Lung Diseases and Idiopathic Pulmonary Fibrosis
- Dysphagia Assessment and Management
- Respiratory and Cough-Related Research
- Vascular Malformations and Hemangiomas
- Pleural and Pulmonary Diseases
- Child Nutrition and Feeding Issues
- Nematode management and characterization studies
- Airway Management and Intubation Techniques
Hospital for Sick Children
2016-2025
University of Toronto
2015-2024
SickKids Foundation
2015-2024
Public Health Ontario
2024
Respiratory Clinical Trials
2009-2023
Great Ormond Street Hospital
2009-2023
University College London
2009-2023
University of Alabama at Birmingham
2023
Muscular Dystrophy Canada
2018-2023
Translational Research Institute
2023
Increasing the activity of defective cystic fibrosis transmembrane conductance regulator (CFTR) protein is a potential treatment for fibrosis.
Cystic fibrosis is a life-limiting disease that caused by defective or deficient cystic transmembrane conductance regulator (CFTR) protein activity. Phe508del the most common CFTR mutation.We conducted two phase 3, randomized, double-blind, placebo-controlled studies were designed to assess effects of lumacaftor (VX-809), corrector, in combination with ivacaftor (VX-770), potentiator, patients 12 years age older who had and homozygous for mutation. In both studies, randomly assigned receive...
Inert gas washout tests, performed using the single- or multiple-breath technique, were first described over 60 years ago. As measures of ventilation distribution inhomogeneity, they offer complementary information to standard lung function such as spirometry, well improved feasibility across wider age ranges and sensitivity in detection early damage. These benefits have led a resurgence interest these techniques from manufacturers, clinicians researchers, yet detailed guidelines for...
Description: Hereditary hemorrhagic telangiectasia (HHT) is an autosomal dominant disease with estimated prevalence of 1 in 5000 that characterized by the presence vascular malformations (VMs). These result chronic bleeding, acute hemorrhage, and complications from shunting through VMs. The goal Second International HHT Guidelines process was to develop evidence-based consensus guidelines for management prevention HHT-related symptoms complications. Methods: were developed using AGREE II...
Rationale Antibiotic therapy for early Pseudomonas aeruginosa infection in patients with cystic fibrosis (CF) is effective, but the optimal therapeutic regimen and duration treatment remains unclear. The EarLy Inhaled Tobramycin Eradication (ELITE) study was designed to assess efficacy safety of two regimens (28 56 days) tobramycin inhalation solution (TIS) 300 mg/5 ml twice daily onset P CF. Methods In this open-label randomised multicentre study, CF (aged ≥6 months) were treated 28 days...
It is unknown what proportion of long-term lung function decline in cystic fibrosis (CF) explained by pulmonary exacerbations. The aim this study was to determine how exacerbations requiring hospitalisation contribute the course CF disease. This a retrospective cohort study. primary outcome rate forced expiratory volume 1 s (FEV(1)) % predicted. Out 851 subjects, 415 (48.8%) subjects had ≥ exacerbation. After adjustment for confounders, annual FEV(1) those without an exacerbation 1.2% per yr...
The cystic fibrosis (CF) modulator drug, elexacaftor/tezacaftor/ivacaftor (ETI), proved highly effective in controlled clinical trials for individuals with at least one F508del allele, which occurs 85% of people CF.
Rationale: The triple-combination regimen elexacaftor/tezacaftor/ivacaftor (ELX/TEZ/IVA) was shown to be safe and efficacious in children aged 6 through 11 years with cystic fibrosis at least one F508del-CFTR allele a phase 3, open-label, single-arm study. Objectives: To further evaluate the efficacy safety of ELX/TEZ/IVA age heterozygous for F508del minimal function CFTR mutation (F/MF genotypes) randomized, double-blind, placebo-controlled 3b trial. Methods: Children were randomized...